A Study to Evaluate the Efficacy and Safety of OTX-TKI (Axitinib Implant) in Participants With Non-Proliferative Diabetic Retinopathy

NCT07235085 | Recruiting Phase 3 FDA Drug

• 1 other identifier: OTX-TKI-2025-NPDR-302
• Study Type: interventional
• Target: 930
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this trial is to study the safety and effectiveness of OTX-TKI (axitinib intravitreal hydrogel) for the treatment of Non-Proliferative Diabetic Retinopathy. OTX-TKI is an intravitreal hydrogel embedded with axitinib. When the OTX-TKI hydrogel is administered into the vitreous cavity of the eye, the hydrogel begins to slowly break down, which allows the axitinib to be slowly released over time. This clinical trial is comparing OTX-TKI to a "sham" injection procedure. The sham injection is a mock injection procedure, but nothing will actually be inserted in the eye as there is no needle on the sham injector. Only one eye ("study eye") will be treated with study treatment.

Conditions

Non-Proliferative Diabetic Retinopathy

Keywords

Non-Proliferative Diabetic Retinopathy

Outcome Measures

Primary Outcomes (1)

• DRSS 2-step change status at Week 52 from baseline in the study eye (≥ 2 step improvement, ≥ 2-step worsening, < 2-step change in either direction)

  The Diabetic Retinopathy Severity Scale (DRSS) measures the severity of the diabetic retinopathy in patients by grading retinal images obtained by color fundus photography. Severity is determined by the presence of specific signs.

  52 weeks

Study Arms (3)

OTX-TKI Q52W (single dose)

EXPERIMENTAL

OTX-TKI 0.45 mg via intravitreal (IVT) administration at Day 1 and sham procedure at Week 24

Drug: Single intravitreal injection of axitinib hydrogel implant followed by a mock (sham) injection procedure at Week 24

OTX-TKI Q24W (2 doses)

EXPERIMENTAL

OTX-TKI 0.45 mg via IVT administration at Day 1 and Week 24

Drug: Intravitreal injection of axitinib hydrogel implant followed by a second intravitreal injection of the axitinib implant at Week 24

Sham Q24W (control)

SHAM COMPARATOR

Sham procedure at Day 1 and Week 24

Other: Sham/mock procedure of intravitreal injection followed by a second sham/mock procedure at Week 24

Interventions

Single intravitreal injection of axitinib hydrogel implant followed by a mock (sham) injection procedure at Week 24 DRUG

OTX-TKI 0.45 mg is a dried polyethylene glycol (PEG)-based hydrogel containing dispersed drug particles of the small molecule axitinib, a tyrosine kinase inhibitor (TKI).

OTX-TKI Q52W (single dose)

Intravitreal injection of axitinib hydrogel implant followed by a second intravitreal injection of the axitinib implant at Week 24 DRUG

OTX-TKI 0.45 mg is a dried polyethylene glycol (PEG)-based hydrogel containing dispersed drug particles of the small molecule axitinib, a tyrosine kinase inhibitor (TKI).

OTX-TKI Q24W (2 doses)

Sham/mock procedure of intravitreal injection followed by a second sham/mock procedure at Week 24 OTHER

sham/mock intravitreal injection procedure

Sham Q24W (control)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female who is at least 18 years of age at the time of signing the informed consent form (ICF)
• History of or newly diagnosed with type 1 or 2 diabetes mellitus and have moderately severe to severe NPDR (DRSS levels 47 or 53), confirmed by the Central Reading Center (CRC) based on the images obtained at the Screening visit
• BCVA Early Treatment Diabetic Retinopathy Study (ETDRS) letter score of ≥ 69 letters (approximate Snellen equivalent of 20/40 or better) in the study eye
• Willing and able to comply with clinic visits and study-related procedures
• Provide signed informed consent

You may not qualify if:

• Presence of center-involved diabetic macular edema (CI-DME) defined per protocol via optical coherence tomography (SD-OCT) in the study eye, obtained at the Screening visit
• Evidence of a rhegmatogenous retinal detachment or visually significant/severe epiretinal membrane, vitreomacular traction syndrome, macular hole, tear of the retinal pigment epithelium in the macula, or other macular pathology in the study eye deemed visually significant by the Investigator
• In the study eye, any panretinal photocoagulation (PRP) treatment prior to baseline, or received focal or grid laser photocoagulation within 1000 microns of the central subfield of the macula within 6 months prior to baseline
• IVT anti-VEGF treatment in the study eye within 6 months or port delivery system (PDS) with ranibizumab in the study eye at any time prior to baseline (Day 1)

Sponsors & Collaborators

• Ocular Therapeutix, Inc.: lead

Study Sites (1)

Cumberland Valley Retina Consultants

Hagerstown, Maryland, 21740, United States

RECRUITING

MeSH Terms

Interventions

salicylhydroxamic acid

Study Officials

• Allen Hu, MD

  Cumberland Valley Retina Consultants

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Clinical Project Manager

CONTACT

7813574000; clinicalaffairs@ocutx.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 14, 2025
• First Posted: November 19, 2025
• Study Start: November 17, 2025
• Primary Completion (Estimated): March 1, 2027
• Study Completion (Estimated): March 1, 2027
• Last Updated: March 16, 2026

Record last verified: 2026-03

Locations

US (1)